Escudero-Saiz Víctor J, Gonzalez Ángela, García-Herrera Adriana, Larque Ana B, Bomback Andrew S, Morantes Laura, Martínez-Chillarón Marta, Ollé Júlia, Guillén Elena, Xipell Marc, Molina-Andújar Alicia, Rodríguez Diana, Cuadrado Elena, Cacho Judit, Arana Carolt, Esforzado Núria, Bastida Carla, Poch Esteban, Diekman Fritz, Cucchiari David, Quintana Luis F, Blasco Miquel
Nephrology and Kidney Transplantation Department, National Reference Center on Complex Glomerular Disease (CSUR), IDIBAPS, Hospital Clínic, University of Barcelona, Barcelona, Spain.
Pathology Service, Hospital Clínic Barcelona, Barcelona, Spain.
Kidney Med. 2024 Apr 12;6(6):100823. doi: 10.1016/j.xkme.2024.100823. eCollection 2024 Jun.
C3 glomerulopathy is a rare disease caused by fluid phase dysregulation of the alternative complement pathway. Currently, treatment depends on clinical and histological severity and includes nephroprotection, unspecific immunosuppression, and terminal complement blockers (C5), without having an etiological treatment approved. C3 glomerulopathy has high recurrence rates after kidney transplantation with a high risk of graft loss. Fortunately, new molecules are being developed that specifically target the proximal alternative complement pathway, such as iptacopan, a factor B inhibitor that showed promising results in native kidneys and cases of transplant recurrence in a phase 2 clinical trial. We present 2 "real-world" cases of C3 glomerulopathy recurrence in kidney allografts treated with iptacopan, with initial excellent clinical response and safety profile, especially with early introduction. We also present follow-up biopsies that showed no C3 deposition during factor B inhibition. Our cases suggest that proximal blockade of the alternative complement pathway can be effective and safe in the treatment of C3 glomerulopathy recurrence in kidney transplantation, bringing other questions such as dual blockade (eg, in C3 and C5), the optimal patient profile to benefit from factor B inhibition or treatment duration and its potential use in other forms of membranoproliferative glomerulonephritis (eg, immune complex-mediated).
C3肾小球病是一种由替代补体途径液相调节异常引起的罕见疾病。目前,治疗取决于临床和组织学严重程度,包括肾保护、非特异性免疫抑制和终末补体阻滞剂(C5),但尚无获批的病因治疗方法。C3肾小球病在肾移植后复发率高,移植肾丢失风险大。幸运的是,正在研发特异性靶向近端替代补体途径的新分子,如iptacopan,一种B因子抑制剂,在2期临床试验中,其在天然肾脏和移植复发病例中显示出了有前景的结果。我们报告了2例接受iptacopan治疗的同种异体肾移植中C3肾小球病复发的“真实世界”病例,初始临床反应和安全性良好,尤其是早期应用时。我们还展示了随访活检结果,显示在抑制B因子期间无C3沉积。我们的病例表明,近端阻断替代补体途径在治疗肾移植中C3肾小球病复发方面可能有效且安全,这也引发了其他问题,如双重阻断(如C3和C5)、从B因子抑制中获益的最佳患者特征、治疗持续时间以及其在其他形式的膜增生性肾小球肾炎(如免疫复合物介导的)中的潜在应用。